Ocugen Advances to Second Dosing Cohort in Phase 1 Trial of OCU200 for Diabetic Macular Edema

Ocugen announced that the DSMB overseeing the ongoing Phase 1 study of OCU200 has approved the initiation of dosing in the second patient cohort. This decision follows successful administration and safety review of the first cohort, which began dosing in early 2025. Importantly, no serious adverse events related to the investigational drug have been reported to date.

OCU200 is a first-in-class fusion protein that combines two human proteins—tumstatin and transferrin—and is designed to be administered via intravitreal injection. Its therapeutic potential spans multiple retinal diseases, including:

       • Diabetic macular edema (DME)

       • Diabetic retinopathy (DR)

       • Wet age-related macular degeneration (wet AMD)

Trial Design and Next Steps

The Phase 1 study is structured as a multicenter, open-label, dose-escalation trial, involving three dose levels:

       • Low dose: 0.025 mg

       • Medium dose: 0.05 mg

       • High dose: 0.1 mg

Each patient will receive two intravitreal injections spaced six weeks apart and will be followed for up to six months to monitor safety outcomes.

Dr. Huma Qamar, Chief Medical Officer at Ocugen, expressed optimism regarding the drug’s profile:

“It is encouraging that we have successfully completed dosing in the low dose cohort for OCU200, a novel biologic that has a very favorable safety and tolerability profile,” said Dr. Qamar. “There remains a considerable unmet medical need for the 30% to 40% of DME patients who do not respond to current anti-VEGF therapies. OCU200 holds the promise of potentially benefiting all DME, diabetic retinopathy, and wet AMD patients.”

Looking Ahead

Ocugen aims to complete the full Phase 1 study by the second half of 2025, with interim safety and efficacy updates expected throughout the year. These early signals will guide the development pathway and further clinical evaluation of OCU200 across broader retinal disease indications.